uniQure's AMT-130 Data Release: Catalyst for Accelerated Approval and Stock Upside Potential

Leerink Partners analyst Joseph Schwartz has maintained a Buy rating for uniQure's (QURE) stock, citing potential accelerated approval and stock upside for the company's AMT-130 in Huntington's disease. Schwartz believes the upcoming data release in September 2025 could provide strong evidence for accelerated approval, leading to a commercial launch and substantial upside potential. Mizuho Securities has also upgraded the stock to a Buy with a $30 price target.

ClearPoint Neuro Inc. (CLPT) reported its Q2 2025 financial results, showing a 17% increase in total revenue to $9.2 million, compared to $7.9 million in Q2 2024 [1]. The company's biologics and drug delivery revenue increased by 10% to $4.7 million, while neurosurgery navigation revenue surged by 33% to $3.4 million. Capital equipment and software revenue also grew by 11% to $1 million. However, the gross margin decreased to 60% from 63% in Q2 2024, primarily due to higher excess and obsolete inventory reserves.

Operating expenses rose by 16% to $11.2 million, driven by a 23% increase in research and development costs and personnel expenses. Despite the revenue growth, the company experienced an operational cash burn of $2.6 million in Q2 2025, similar to the previous year. The company's cash and cash equivalents stood at $41.5 million as of June 30, 2025, up from $20.1 million at December 31, 2024.

ClearPoint Neuro Inc. has expanded its global presence, with technology now available in over 100 centers worldwide and regulatory clearance in 36 countries. The introduction of new products like the 3.0 ClearPoint ICT navigation software and Prism laser therapy system has been well-received, contributing to increased hospital efficiency. The company is actively supporting over 60 biopharma partners in cell and gene therapy development, with several partners progressing to regulatory trials and expedited FDA reviews.

uniQure's (QURE) AMT-130 gene therapy for Huntington's disease (HD) is on track for FDA accelerated approval via a streamlined regulatory pathway using natural history data as a control [2]. Phase I/II trials showed statistically significant disease progression slowing and 11% CSF NfL reduction, with 3-year safety data expected in late 2025. Mizuho upgraded QURE to Outperform with a $30 price target, citing $2.5B peak sales potential, strong biomarker validation, and reduced Phase III failure risk due to FDA's biomarker endpoint acceptance. Leerink Partners analyst Joseph Schwartz has maintained a Buy rating for QURE, citing potential accelerated approval and stock upside for AMT-130. Schwartz believes the upcoming data release in September 2025 could provide strong evidence for accelerated approval, leading to a commercial launch and substantial upside potential. Mizuho Securities has also upgraded the stock to a Buy with a $30 price target.

References:
[1] https://finance.yahoo.com/news/clearpoint-neuro-inc-clpt-q2-071230689.html
[2] https://www.ainvest.com/news/uniqure-amt-130-road-accelerated-fda-approval-strategic-buy-opportunity-gene-therapy-2508/